The Next Wave: Pharm Exec's 2011 Pipeline Report

Like a mermaid or a minotaur, pharma's 2011 pipeline is a shape-shifting creature—a hybrid of primary care and specialty products; mass-market molecules and orphan-disease biologics; novel vaccines for neglected diseases that may save hundreds of thousands of lives at cost; and follow-on oncologics that may add a few months of survival with six-digit price tags for the few who can afford them. New forms of pharma life are rising out of the defunct blockbuster model, but they remain in morphing mode.

The 42 compounds in Pharm Exec's "2011 Pharma Pipeline Report" reflect this evolutionary moment. The list of firsts, of significant advances and downright breakthroughs, is unusually promising. New classes of HIV-inspired protein inhibitors are set to increase the cure rate in Hepatitis C by 50 percent. In multiple sclerosis, the advent of new oral therapies and new approaches may dramatically cut disease progression. Patients with lupus will have the first new treatment in half a century, and better, safer bloodthinners will finally lay old warhorse warfarin to rest. The vaccine pipeline heralds first-ever prevention against such widely varied threats as malaria, MRSA bacteria, and anthrax. And in cancer a few new treatments have hit the bull's-eye on new targets and pathways, including previously untreatable triple-negative breast cancer and advanced melanoma, while a small group of non-small-cell lung cancer patients may have a Gleevec-like success story. Meantime, the anti-CETP inhibitors have risen from the grave, as several competing molecules, much-tweaked for safety, race to become the first-in-class good-cholesterol gooser—and rake in Lipitor-sized profits.

Drug Index

This list is testimony to a level of innovation any industry would be proud of. But there are some telling absences. CNS remains the black box of R&D. With Alzheimer's a looming public health crisis, the field is littered with Phase III failures, and the fate of the final two or three megablockbuster-to-be is in grave danger. Obesity resists pharmaceutical intervention. Even diabetes, however well treated, still lacks a truly disease-modifying drug.

Inevitably, the learning curve for pharma will remain steep for the foreseeable future. Only yesterday, it seems, the industry finally grasped that me-toos and marginal improvements no longer cut it. Drugmakers went mad for targets, screening and acquiring mountains of compounds in diseases they knew well and those they did not.

"The next innovation for pharma will come from a more comprehensive approach to disease," says Mike Wokash, a veteran of Big Pharma who blogs at
http://Pharmareform.com/. "Too many of the big players are still looking for a product in a market for next year rather than looking to make a real impact on a disease in five or 10 years." Now, with comparative effectiveness and price pressures in the offing, regulators, payers, and consumers are all demanding better value, guaranteed efficacy, and proven outcomes—and for pharma, chasing innovation demands more investment of all kinds than ever before.